FDA Approves First Treatment Option for Aggressive Sarcoma Subtype

This marks the only available treatment option approved by the FDA for adults with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor.

The Food and Drug Administration (FDA) has approved the mTOR inhibitor Fyarro (nab-sirolimus) for the treatment of adults with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor.

This marks the first and only FDA-approved treatment for patients with this disease, which is a rare soft-tissue sarcoma, according to a release from the company. Between 100 and 300 new patients per year are diagnosed with a perivascular epithelioid cell tumor, which can appear in any body site including the lung, uterus, retroperitoneum, kidney, liver, gastrointestinal tract and genitourinary tract. It is diagnosed more often in women.

“We are thrilled to have received full FDA approval of Fyarro,” said Neil Desai, founder, chief executive officer and president of Aadi, the manufacturer of the drug, in the release. “The approval of Fyarro is a momentous event not just for Aadi, but, importantly, for advanced malignant (perivascular epithelioid cell tumor) patients.”

This FDA approval was based on findings from the phase 2 AMPECT trial, which demonstrated that patients had an overall response rate (percentage of patients with a partial or complete response to treatment) of 39%, with two patients achieving a complete response after long-term follow-up, according to the release. During a median follow-up of 36 months, the median duration of response had not been reached and ranged from 5.6 months to greater than 55.5 months.

In addition, results from the AMPECT trial demonstrated that 92% of patients had a response to the treatment lasting longer than or equal to six months, 67% with a response lasting greater than or equal to 12 months and 58% had a response lasting greater than or equal to two years.

“The approval of Fyarro, the first approved drug for advanced malignant (perivascular epithelioid cell tumor), an aggressive sarcoma with a poor prognosis and few treatment options, will provide physicians with a new weapon for treating patients with this rare disease,” said Dr. Andrew Wagner, a senior oncologist at Dana-Farber Cancer Institute and principal investigator of the AMPECT trial, in the release. “In our AMPECT trial, Fyarro demonstrated durable responses in mTOR inhibitor-naïve patients with locally advanced unresectable or metastatic (perivascular epithelioid cell tumor), with an acceptable and manageable safety profile.”

Prescribing information for Fyarro has several warnings and precautions related to myelosuppression (decrease in bone marrow), stomatitis (inflamed, sore mouth), hypokalemia (low potassium level), infections, hyperglycemia (high blood sugar), hemorrhage (bleeding), interstitial lung disease (scarring of the lungs) and hypersensitivity reactions.

“Patients living with locally advanced or metastatic (perivascular epithelioid cell tumor) are in urgent need of new treatment options,” said Dr. Robert G. Maki, clinical director of the sarcoma program and professor of medicine at the University of Pennsylvania, in the release. “The approval of Fyarro is a significant advancement for treating patients with this disease. Treating sarcoma patients in my practice, I have seen the need for a therapy that addresses the specific molecular alterations of advanced malignant (perivascular epithelioid cell tumor). ... I am pleased to have Fyarro as a new therapeutic option to offer my advanced malignant (perivascular epithelioid cell tumor) patients.”

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